CN101952434B - 使用合成的聚合物递送对基因沉默有活性的核酸的工具 - Google Patents

使用合成的聚合物递送对基因沉默有活性的核酸的工具 Download PDF

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Publication number
CN101952434B
CN101952434B CN200880125788.1A CN200880125788A CN101952434B CN 101952434 B CN101952434 B CN 101952434B CN 200880125788 A CN200880125788 A CN 200880125788A CN 101952434 B CN101952434 B CN 101952434B
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sirna
compositions
rna
transfection
cells
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CN101952434A (zh
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A·阿迪布
P·埃尔巴彻
F·斯多克
N·哈夫迪
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Polyplus Transfection SA
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Polyamides (AREA)
CN200880125788.1A 2007-12-13 2008-12-12 使用合成的聚合物递送对基因沉默有活性的核酸的工具 Active CN101952434B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1335807P 2007-12-13 2007-12-13
US61/013,358 2007-12-13
PCT/IB2008/055256 WO2009074970A2 (en) 2007-12-13 2008-12-12 Means for delivery of nucleic acids active for gene silencing using synthetic polymers

Publications (2)

Publication Number Publication Date
CN101952434A CN101952434A (zh) 2011-01-19
CN101952434B true CN101952434B (zh) 2016-11-16

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CN200880125788.1A Active CN101952434B (zh) 2007-12-13 2008-12-12 使用合成的聚合物递送对基因沉默有活性的核酸的工具

Country Status (8)

Country Link
US (1) US20100297756A1 (enExample)
EP (1) EP2229445B1 (enExample)
JP (1) JP5802389B2 (enExample)
KR (1) KR101590652B1 (enExample)
CN (1) CN101952434B (enExample)
CA (1) CA2708354C (enExample)
IL (1) IL206310A (enExample)
WO (1) WO2009074970A2 (enExample)

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EP2552460A1 (en) 2010-03-29 2013-02-06 Universite De Strasbourg Polymers for delivering molecules of interest
KR101223483B1 (ko) * 2010-09-10 2013-01-17 한국과학기술연구원 전하결합 및 생분해성 공유결합으로 동시에 연결된 고분자―siRNA 나노입자 전달체
KR101497668B1 (ko) * 2012-09-28 2015-03-03 한양대학교 산학협력단 전신 순환을 위한 짧은 간섭 rna 유전자 전달체
WO2014051318A2 (ko) * 2012-09-28 2014-04-03 한양대학교 산학협력단 전신 순환을 위한 짧은 간섭 rna 유전자 전달체
EP4085930A1 (en) 2012-10-05 2022-11-09 BioNTech Delivery Technologies GmbH Hydroxylated polyamine derivatives as transfection reagents for rna
CN104703629B (zh) 2012-10-08 2020-12-01 生物技术传送科技有限责任公司 作为转染试剂的羧化多胺衍生物
CN103087314B (zh) * 2013-02-01 2015-02-18 同济大学 生物可降解聚氨酯基因转染试剂的制备方法
CN107805643B (zh) * 2017-09-15 2020-12-08 四川大学 靶向抑制沙门氏菌耐药外排泵基因acrA的siRNA-DNA纳米系统及其制备方法
BR112022002149A2 (pt) * 2019-08-05 2022-04-19 Polyplus Transfection Composição adequada para transfectar uma molécula de ácido nucleico em uma célula, métodos para transfecção in vitro ou ex vivo de células vivas e para a produção e usos in vitro ou ex vivo da composição
DE102020114183B4 (de) 2020-05-27 2022-07-14 Universität Leipzig Dezernat Forschung und Transfer Mittel zur transfektion von nukleinsäuren in zellen
CN112195193A (zh) * 2020-09-30 2021-01-08 上海交通大学 单链dna在基因转染中的应用
US20240327830A1 (en) * 2022-02-28 2024-10-03 Northwestern University Dise-inducing srna-polyplexes and srna-lipopolyplexes and methods of using the same to treat cancer

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US7427394B2 (en) * 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US7601367B2 (en) * 2002-05-28 2009-10-13 Mirus Bio Llc Compositions and processes using siRNA, amphipathic compounds and polycations
EP1546173A4 (en) * 2002-08-06 2006-05-31 Intradigm Corp METHOD FOR DOWNSTREGULATING THE EXPRESSION OF TARGET GENES IN VIVO BY INCLUDING INTERFERING RNA
JP2006517793A (ja) * 2003-01-31 2006-08-03 ノバルティス アクチエンゲゼルシャフト スクリーニングアッセイ
JP2007502129A (ja) * 2003-05-09 2007-02-08 ユニヴァーシティ オヴ ピッツバーグ オヴ ザ コモンウェルス システム オヴ ハイアー エデュケーション 短鎖干渉rnaライブラリーならびに合成および使用の方法
SG190613A1 (en) * 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
WO2005059111A2 (en) * 2003-12-17 2005-06-30 The Trustees Of Columbia University In The City Of New York DELIVERY OF DNA OR RNA VIA GAP JUNCTIONS FROM HOST CELLS TO TARGET CELLS AND A CELL-BASED DELIVERY SYSTEM FOR ANTISENSE OR SiRNA
JP2008527993A (ja) * 2005-01-24 2008-07-31 アヴァリス・アクチエボラーグ 特異性及び送達の改善のためのsiRNA分子、shRNA分子又はアンチセンス分子及び機能的実体を含む複合体
US20100310670A1 (en) * 2005-09-02 2010-12-09 Takeda Pharmaceutical Company Limited Sustained-release microsphere containing short chain deoxyribonucleic acid or short chain ribonucleic acid and method of producing the same
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Munetaka kunishima 等.Formation of carboxamides by direct condensation of carboxylic acids and amines in alcohols using a new alcohol- and water- soluble condensing agent:DMT-MM.《Tetrahedron》.2001,第57卷(第8期),1551-1558. *
张猛.末端修饰的PAMAM非病毒基因治疗载体的研究.《中国优秀博硕士学位论文全文数据库(硕士) 医药卫生科技辑》.2007,(第6期), *
房宝英 等.siRNA及其导入体内外方法的研究进展.《国际病理科学与临床杂志》.2006,第27卷(第1期),44-47. *

Also Published As

Publication number Publication date
IL206310A (en) 2013-07-31
KR101590652B1 (ko) 2016-02-18
CN101952434A (zh) 2011-01-19
US20100297756A1 (en) 2010-11-25
KR20100105674A (ko) 2010-09-29
JP5802389B2 (ja) 2015-10-28
WO2009074970A3 (en) 2009-10-29
EP2229445B1 (en) 2012-12-05
JP2011505826A (ja) 2011-03-03
EP2229445A2 (en) 2010-09-22
WO2009074970A2 (en) 2009-06-18
IL206310A0 (en) 2010-12-30
CA2708354C (en) 2018-12-11
CA2708354A1 (en) 2009-06-18

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